Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...

A new gene editor may soon open the door to gene therapies for a wider array of diseases. Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of congenital deafness. When you purchase through links on our site, we may earn an ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

This story is the first in an occasional series following Louisiana’s first patients to receive gene therapy for sickle cell disease. On the third floor of Manning Family Children’s Hospital in New ...

Maoli Duan does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond their ...